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7 days ago
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BioCryst Highlights Real-world Data Showing ORLADEYO® (berotralstat) Consistently Reduces HAE Attacks Across All Ages
New data shared at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports berotralstat use at all stages of life RESEARCH TRIANGLE PARK, N.C., May 30, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data which highlights the reduction in the percentage of days with hereditary angioedema (HAE) symptoms among young children initiating berotralstat in the APeX-P trial, and the broad safety and efficacy outcomes observed across all age groups of patients taking ORLADEYO to prevent HAE attacks. 'These data underscore the potential of our oral, once-daily prophylactic treatment to deliver consistent, meaningful benefits for people living with HAE across all stages of life. From young children to adolescents and adults, ORLADEYO continues to show strong efficacy and tolerability, helping patients experience more days free from attacks. These clinical and real-world results give me confidence that we can continue to have a very positive impact on advancing care for all those living with HAE, regardless of their age,' said Helen Thackray, chief research and development officer at BioCryst. The following three studies are being presented at the 14th C1-Inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary from May 29–June 1, 2025. Reduction in Percentage of Days with Angioedema Symptoms Among Young Children Initiating Berotralstat – Interim Results from ApeX-P Oral Presentation - Session IV - Saturday, May 31 - 10:30 – 12:45 (12:00-12:15) The ongoing open-label APeX-P study is evaluating berotralstat in children aged 2 to 11 years with HAE. There were four weight-based cohorts. Cohort 1 from these interim results received 150 mg capsules daily, while cohorts 2–4 received daily oral granule doses of 108, 96, and 78 mg, respectively. The median age was 8 years (range: 3–11), with disease onset typically between ages 2–6 years. Prior to enrollment, patients reported a mean (SD) of 18.0 (20.5) missed school days annually due to HAE. While on berotralstat, at least half of patients were attack-free each month. The percentage of days with angioedema symptoms dropped from a mean (SD) of 11 percent (9.4 percent) during the standard-of-care period, measured pre-study, to 4.0 percent (4.7 percent) over 12 weeks, with this improvement sustained up to 48 weeks. The most common treatment emergent adverse events (TEAEs) were nasopharyngitis, upper respiratory tract infection and headache. These interim findings suggest berotralstat may help children with HAE experience fewer symptoms and a more normal daily life. Berotralstat for the Prevention of Hereditary Angioedema Attacks: Results from the Italian Expanded Access Program Oral Presentation - Session VI- Sunday, June 1 - 08:00 – 09:30 (09:00-09:15) In the Italian expanded access program (EAP), 22 patients received berotralstat free of charge until commercial availability, with an additional 12 weeks of treatment to ensure continuity. Safety was monitored throughout, and data on breakthrough attacks, disease control, and quality of life (QoL) were collected. Among 12 patients with available data, median (mean) Angioedema Control Test (AECT) scores improved from 11.5 (12.8) at baseline to 13.0 (14.0) at month three and 16.0 (14.8) at month six, indicating better disease control. Median (mean) AE-QoL total scores decreased from 45.0 (42.3) at baseline to 40.0 (37.6) at month three and 36.2 (34.0) at month six, reflecting clinically meaningful improvements in QoL. All 15 patients still enrolled at program closure transitioned to commercially available ORLADEYO. The treatment was associated with a consistently low HAE attack rate, improved disease control and a safety profile consistent with clinical trial findings. Tolerability and Effectiveness of Berotralstat for Prophylaxis in Adolescents with Hereditary Angioedema: subgroup analysis of the Berolife Study Oral Presentation - Session VI - Sunday June 1 – 08:00-9:30 (08:30-08:45) The Berolife study, a real-world observational study in France, assessed the tolerability and effectiveness of berotralstat in patients with HAE, including a subgroup of adolescents (aged 12–17) who received 150 mg once daily. The mean (SD) baseline attack rate was 1.8 (1.1) attacks/month (median: 1.8), based on the six months prior to enrolment. After six months of treatment, the median monthly attack rate decreased to 0.55 (from 2.25 at baseline), with similar reductions sustained at 12 and 18 months. TEAEs in adolescents were consistent with those seen in the overall Berolife population and in the APeX-2 and APeX-S clinical trials. These findings support berotralstat as an effective and well-tolerated long-term prophylactic option for adolescents with HAE. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety InformationINDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of use The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATION An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John's wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or Please see full Prescribing Information. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance and effectiveness. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst's ability to successfully implement or maintain its commercialization plans for ORLADEYO; interim results of a clinical trial do not necessarily predict final results; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; and BioCryst's ability to successfully manage its growth and compete effectively. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@ Niamh Lyons +353 87 7745000nlyons@ in to access your portfolio
Yahoo
16-05-2025
- Health
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BioCryst Presents New Real-world Evidence Showing Significant and Sustained Reductions in HAE Attack Rates in Adolescents and People with Severe HAE Following Initiation of ORLADEYO® (berotralstat)
RESEARCH TRIANGLE PARK, N.C., May 16, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence on the use of oral, once-daily ORLADEYO® (berotralstat) in adolescents and people with severe HAE showing significant and sustained reductions in HAE attack rates through 18 months of follow-up after beginning treatment with ORLADEYO in both patient populations. The real-world evidence was presented in two posters at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR), which is being held in Montreal from May 13-16, 2025. 'The outcomes detailed in these posters show how ORLADEYO is making a difference for people living with HAE, in particular those with very severe disease and those who are adolescents. These two groups experienced far fewer attacks per month compared to baseline after starting ORLADEYO. These kinds of real-world results should give physicians as well as their HAE patients the additional confidence to improve control of their attacks,' said Dr. Raffi Tachdjian, associate clinical professor of medicine & pediatrics, division of allergy & clinical immunology, David Geffen School of Medicine, University of California Los Angeles. Significant and sustained reductions in attack rates after ORLADEYO initiation The results presented in two posters at ISPOR 2025 were from a retrospective pre-post study using outcomes collected from BioCryst's sole-source pharmacy from December 15, 2020, to January 8, 2024. The poster 'Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients with C1 Inhibitor Deficiency (Type I/II) and ≥8 Attacks/month' (#PCR182) detailed findings from 56 U.S. patients with HAE with C1-inhibitor deficiency (HAE-C1-INH) who received ORLADEYO. Patients experienced significantly lower HAE attack rates while on ORLADEYO in each 90-day follow-up period (1.24-1.90 attacks/month) compared to baseline (7.78-8.23 attacks/month). Patients experienced significantly fewer HAE attacks per month following ORLADEYO initiation during every 90-day follow-up period relative to baseline, including: 6.25 fewer attacks/month (95 percent confidence period (CI): [5.63, 6.87]; p<0.001) at 12 months (days 271-360) 6.43 fewer attacks/month (95 percent CI: [5.78, 7.09]; p<0.001) at 18 months (days 451-540) The poster 'Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Adolescents' (#PCR132) highlighted outcomes reported from 99 U.S. patients with HAE aged 12-17 years who received ORLADEYO. Patients had significantly lower HAE attack rates while on ORLADEYO during each 90-day follow-up period (0.36-0.76 attacks/month) compared to the mean baseline rate (2.07-2.30 attacks/month). Compared to baseline, adolescents experienced statistically significant and sustained reductions in HAE attack rates after ORLADEYO initiation during each 90-day follow-up period, including: 1.56 fewer attacks/month (95 percent CI: [0.89, 2.23]; p<0.001) at 12 months (days 271-360) 1.85 fewer attacks/month (95 percent CI: [1.12, 2.58]; p<0.001) at 18 months (days 451-540) 'We continue to generate evidence from real-world use of our oral, once-daily prophylactic therapy for HAE that supports its effectiveness in a wide range of people with HAE. Here, we show that ORLADEYO is having a positive impact on attack reduction for younger people and those with severe disease. These additional findings further underscore that ORLADEYO works well for many patients with HAE, regardless of their attack severity, age or other aspects,' said Dr. Donald S. Fong, chief medical officer of BioCryst. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATIONAn increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John's wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or Please see full Prescribing Information. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance and effectiveness. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst's ability to successfully implement or maintain its commercialization plans for ORLADEYO; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; and BioCryst's ability to successfully manage its growth and compete effectively. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
06-05-2025
- Business
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BioCryst Pharmaceuticals Inc (BCRX) Q1 2025 Earnings Call Highlights: Strong ORLADEYO Sales ...
Total Revenue: $145.5 million for the quarter. ORLADEYO Revenue: $134.2 million, with $120.2 million from the US. Operating Expenses (excluding stock-based compensation): $102.9 million, up from $93.6 million in the same quarter last year. Operating Profit: $21.2 million for the first quarter of 2025. Net Income: Slightly positive for the quarter. Cash Position: $317 million at the end of the quarter. Debt Reduction: Paid down $75 million, reducing outstanding debt to $249 million. Annual Revenue Guidance for ORLADEYO: Raised to between $580 million and $600 million, representing 33% to 37% growth over last year. Non-GAAP Operating Expense Guidance: $440 million to $450 million for the year. Warning! GuruFocus has detected 6 Warning Signs with BCRX. Release Date: May 05, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) reported a strong first quarter with revenue of $145.5 million, driven by ORLADEYO sales. The company raised its annual revenue guidance for ORLADEYO to between $580 million and $600 million, indicating 33% to 37% growth over the previous year. BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) achieved a significant improvement in the paid rate for ORLADEYO, reaching 84%, which was expected to take three years but was accomplished in four months. The company is on track to be profitable on a full-year basis in 2025, a year earlier than previously planned. BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) paid down $75 million of its debt, reducing outstanding debt to $249 million and saving approximately $23 million in interest payments over the life of the debt. The typical revenue jump in Q2 will be less pronounced than in prior years due to the accelerated revenue capture in Q1. Operating expenses increased to $102.9 million, up from $93.6 million in the same quarter last year, driven by commercial expansion and support activities. The company faces challenges in conducting clinical trials for rare diseases like Netherton syndrome due to the lack of existing therapies and organized patient advocacy. The path to achieving $1 billion in global revenue by 2029 for ORLADEYO is still dependent on capturing more patients and maintaining growth momentum. The pediatric launch of ORLADEYO, while promising, has not yet been quantified in terms of revenue impact, and the uptake dynamics remain uncertain. Q: Can you provide insights into the potential pediatric launch for ORLADEYO and its impact on the market? A: Charles Gayer, Chief Commercial Officer, explained that there are approximately 500 pediatric HAE patients in the US, with at least 200 potentially suitable for prophylactic therapy. The introduction of an oral option like ORLADEYO could change the treatment landscape, as there is no preference for injectables among children. The expectation is that ORLADEYO will become the market leader due to its ease of administration compared to injectables. Q: What are the expectations for the DME program's first readout, and what would be considered a successful outcome? A: Helen Thackray, Chief Research & Development Officer, stated that the focus is on safety, tolerability, and the effect on retinal thickness. The goal is to observe a reduction in retinal edema, with preclinical data suggesting a quick response. A significant reduction in edema would be a positive indicator for the program's success. Q: How did BioCryst achieve such a significant improvement in the paid rate for ORLADEYO, and is this sustainable? A: Charles Gayer highlighted that two-thirds of the improvement was due to the Inflation Reduction Act, which helped Medicare patients afford their prescriptions. The commercial segment also saw improvements due to better preparation and real-world evidence supporting ORLADEYO's efficacy. The current paid rate is expected to be sustainable, with further incremental improvements anticipated. Q: With profitability pulled forward, how does this impact BioCryst's investment strategy, particularly for the early-stage pipeline? A: Jon Stonehouse, CEO, noted that the programs are already fully funded to proceed as quickly as possible. The company is focused on cleaning up the balance sheet, as evidenced by the recent $75 million debt paydown, and will continue to look at similar opportunities as cash flow improves. Q: What are the expectations for the Netherton syndrome program, and what are the key milestones? A: Helen Thackray explained that the program is designed to address the fundamental pathology of Netherton syndrome. The Phase 1 trial will assess drug levels in the epidermis and clinical impact, with initial data expected by year-end. The goal is to see normalization of the skin and reduction in itch, with potential dose escalation if necessary. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
21-04-2025
- Business
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BioCryst to Report First Quarter 2025 Financial Results on May 5
RESEARCH TRIANGLE PARK, N.C., April 21, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will report its first quarter 2025 financial results on Monday, May 5, 2025. BioCryst management will host a conference call and webcast at 8:30 a.m. ET that day to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. BCRXW Contact:John Bluth+1 919 859 7910jbluth@
Yahoo
02-04-2025
- Business
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BioCryst to Present at Upcoming Investor Conference
RESEARCH TRIANGLE PARK, N.C., April 02, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company plans to present at the 24th Annual Needham Virtual Healthcare Conference on Wednesday, April 9, 2025, at 9:30 a.m. ET. Link to the live audio webcast and replay of the presentation may be accessed in the Investors & Media section of BioCryst's website at About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. BCRXW Contact:John Bluth+1 919 859 7910jbluth@ in to access your portfolio
